*All Times Listed are in GMT

8:50 am Chair’s Opening Remarks

SETTING THE SCENE: PROGRESS OF GENE THERAPY DEVELOPMENT IN EUROPE

9:00 am Leveraging Knowledge from an Approved Gene Therapy Program in Europe: Key Learnings

  • Mike Fraser General Manager Europe, Middle East and Africa, Novartis Gene Therapies

Synopsis

  • Understanding the steps needed to deliver a comprehensive package to payors for early access
  • Post-approval, what are the next steps to gain market access, and when’s the right time to prepare?
  • Discussing frameworks that must be in place to ensure sustainable patient access

9:30 am Developing a Comprehensive Translational Strategy to Set up for Preclinical & Clinical Programs

  • Anne Douar Chief Development Officer, Vivet Therapeutics

Synopsis

  • Assessing the importance of establishing effective animals to bridge the gap to the clinic
  • Highlighting immune challenges early on in development and how to combat them
  • Ensuring safety and efficacy data is in line with regulatory expectations

10:00 am In-process control for pDNA production

  • Blaž Bakalar Product Manager, BIA Separations, a Sartorius Company

Synopsis

Global demand for pDNA production is at an all-time high, due to increased need from Gene Therapy ramp-up. pDNA, as an enabling product, is critical in production of mRNA, AAV and other therapeutic vectors. Increasing yield and purity in the production of pDNA is a vital step in meeting such demand. Supporting reliable in-process control during pDNA purification, PATfix pDNA analytical platform is enabling rapid process development and optimization while providing a reliable analytical platform for production runs.

10:30 am Industry Leaders Panel: Delivering Transformative Therapies to Patients in a Complex European Landscape

Synopsis

  • What can the industry do to address safety concerns surrounding gene therapy?
  • Similarities and key differences in a US vs. European market
  • Identifying the biggest bottlenecks in gene therapy and how they are being addressed by the pioneering companies in the space

11:00 am Virtual Speed Networking

Synopsis

Grab a cup of coffee from the comfort of your own kitchen and jump straight into this exclusive virtual speed networking! This session is the ideal opportunity to meet face-to-face (albeit 2D faces) with many of the brightest minds in the industry to establish meaningful business relationships.

MANUFACTURING
Managing Process Development for Effective Outcomes

11:30 am Improving AAV Product Quality & Addressing Viral Clearance

Synopsis

  • Removal of host cell proteins across affinity and anion exchange chromatography
  • Removal of empty capsids across anion exchange chromatography
  • Removal of enveloped viruses across the purification process and evaluation of new viral clearance steps to improve viral safety

12:00 pm Assessing Subpopulations of AAV Vectors More Effectively for Product Characterization

Synopsis

  • Effectively characterizing for full, empty and partially filled capsids
  • How can the ratio of full to empty be further optimised?

12:30 pm Speaking Position TBC

CLINICAL & COMMERCIAL
Establishing Robust Payment Models for Gene Therapy

11:30 am Analysing Effectiveness of Outcome-Based Payment Models so Far in Germany

Synopsis

  • Exploring how companies have negotiated for outcome-based agreements in Europe
  • Working within existing legislation to get reimbursement for patients
  • How will this system need to evolve to accommodate future approvals in the pipeline over the next five years?

12:00 pm Understanding the HTA Perspective on Gene Therapy Pricing & Reimbursement

Synopsis

  • Exploring how prices of potential comparators could influence pricing decisions
  • Understanding how HTAs assess each gene
    therapy and therefore what data is required
  • How to collaborate early on with HTAs

12:30 pm Digital Transformation in Precision Medicine; Addressing Regulatory and Compliance Demands

  • Karri Unruh Director, Product Management, L7 Informatics

Synopsis

  • Digitalization in the space
  • Addressing regulatory and compliance challenges
  • Using a unified platform for automation and data management to meet necessary requirements

1:00 pm | Lunch & Networking

MANUFACTURING
Refining Upstream Manufacturing Processes

2:00 pm Comparing Lentiviral & AAV Manufacturing Considerations

Synopsis

  • Discussing progress in lentiviral analytics and characterization – similarities and
    differences to AAV
  • Considerations when pursuing multiple vector types
  • Making decisions based on disease indication & payload size

2:30 pm An Optimized Platform Process for Large-scale Plasmid Purification

Synopsis

  • Challenges in pDNA manufacturing, Single-Use platform process designed to address the bottlenecks in large scale purification of plasmid.
  • Significance of designing a process with quality as priority.

3:00 pm Panel Discussion: Transitioning Effectively from Clinical to Commercial Manufacturing

Synopsis

  • Optimizing the transition in manufacturing process from clinical to commercial
    manufacturing
  • How do changes in production influence the overall CMC & clinical protocol?
  • Identifying appropriate methods to safeguard manufacturing robustness

CLINICAL & COMMERCIAL
Addressing Gene Therapy Safety in Clinical Development

2:00 pm Regulatory Challenges and Opportunities for Gene Therapy

Synopsis

  • What safety data are the regulators looking for from preclinical data to proceed
    into a clinical trial?
  • Key regulatory interactions with the FDA early on in development
  • Case study: IND filing of a second-generation gene therapy for Hemophilia A

2:00 pm Panel Discussion: Strategic Intelligence: Shifting ‘So What’ Mindsets to ‘Now What’ Mindsets

2:30 pm Addressing Gene Therapy Safety in Clinical Development & Long-Term Follow-Ups

  • Simon Hawkins Senior Clinical Development Director, Orchard Therapeutics

Synopsis

  • Defining measures of safety in clinical trials to meet regulatory expectations
  • Measuring and collating long-term safety data
  • Exploring requirements of post-approval safety and role of pharmacovigilance

3:30 pm | Afternoon Break & Poster session

ENSURING PATIENT-CENTRIC GENE THERAPY DEVELOPMENT AT EVERY STAGE

4:30 pm Progressing Gene Therapies in Ultra Rare Disease: Key Considerations

Synopsis

  • Exploring regulatory development in the context of ultra-rare diseases
  • Discussing clinical development, particularly trial recruitment and suitable comparators
  • Understanding the commercial approach for ultra-rare diseases

5:00 pm Developing Effective Collaborations with Patient Groups across a Fragmented European Landscape

Synopsis

  • Unique dynamics of operating in a European environment and related challenges for patient engagement in gene therapy development and commercialisation
  • Learnings and comparisons from the US & EU experiences
  • Where is Europe leading the way and what might the future look like as more gene therapies advance toward approval?

5:30 pm Panel Discussion: How can Industry Work More Effectively with Patient Groups in Europe?

Synopsis

  • Establishing relationships with a range of advocacy groups, from international to regional
  • Creating an accessible partnership, including considerations of language and cultural differences
  • Managing expectations between different stakeholders from early in development

6:00 pm Chair’s Closing Remarks